JAMA neurology最新文献

{"title":"Fecal Microbiome Transplants For Parkinson Disease.","authors":"Timothy R Sampson","doi":"10.1001/jamaneurol.2024.2293","DOIUrl":"10.1001/jamaneurol.2024.2293","url":null,"abstract":"","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":" ","pages":"911-913"},"PeriodicalIF":20.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141788032","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Combined Physiotherapy and Cognitive Behavioral Therapy for Functional Movement Disorders: A Randomized Clinical Trial.","authors":"Daniel Macías-García, Marta Méndez-Del Barrio, Manuel Canal-Rivero, Laura Muñoz-Delgado, Astrid Adarmes-Gómez, Silvia Jesús, Elena Ojeda-Lepe, Fátima Carrillo-García, Francisco J Palomar, Francisco Javier Gómez-Campos, Juan Francisco Martin-Rodriguez, Benedicto Crespo-Facorro, Miguel Ruiz-Veguilla, Pablo Mir","doi":"10.1001/jamaneurol.2024.2393","DOIUrl":"10.1001/jamaneurol.2024.2393","url":null,"abstract":"<p><strong>Importance: </strong>Functional movement disorders (FMDs) are frequent and disabling neurological disorders with a substantial socioeconomic impact. Few randomized studies have analyzed the effectiveness of combined physiotherapy and psychotherapy in patients' quality of life.</p><p><strong>Objective: </strong>To assess the efficacy of multidisciplinary treatment (physiotherapy plus cognitive behavioral therapy) in FMDs.</p><p><strong>Design, setting, and participants: </strong>This was a parallel, rater-blinded, single-center, randomized clinical trial. Recruitment took place from June 2022 to April 2023, and follow-up visits were performed at months 3 and 5, concluding in October 2023. Participants were recruited from a national referral center for movement disorders: the Movement Disorders Unit from the Hospital Universitario Virgen Rocio in Seville, Spain. Patients had to be 18 years or older with a confirmed FMD diagnosis and capable of giving consent to participate. Patients who did not meet eligibility criteria or refused to participate were excluded. Any uncontrolled psychiatric disorder was considered an exclusion criterion.</p><p><strong>Interventions: </strong>Patients were randomly assigned, in a ratio of 1:1 to multidisciplinary treatment (physiotherapy plus cognitive behavioral therapy), or a control intervention (psychological support intervention).</p><p><strong>Main outcomes and measures: </strong>Primary outcomes: between-group differences in changes from baseline to month 3 and month 5 in patients' quality of life (EQ-5D-5L score: EQ Index and EQ visual analog scale [EQ VAS]; and 36-Item Short-Form Survey Physical Component Summary [SF-36 PCS] and SF-36 Mental Component Summary [MCS]). Linear mixed models were applied, controlling by baseline severity and applying Bonferroni correction.</p><p><strong>Results: </strong>Of 70 patients screened with an FMD, 40 were enrolled (mean [SD] age, 43.5 [12.8] years; age range, 18-66 years; 32 female [80%]; mean [SD] age at FMD onset, 38.4 [12.1] years), and 38 completed all the follow-up visits and were included in the analysis for primary outcomes. Multidisciplinary treatment improved SF-36 PCS with a mean between-group difference at 3 months of 4.23 points (95% CI, -0.9 to 9.4 points; P = .11) and a significant mean between-group difference at 5 months of 5.62 points (95% CI, 2.3-8.9 points; P < .001), after multiple-comparisons adjustment. There were no significant differences in other quality-of-life outcomes such as SF-36 MCS (mean between-group difference at 3 and 5 months: 0.72 points; 95% CI, -5.5 to 7.0 points; P = .82 and 0.69 points; 95% CI, 2.3-8.9 points; P = .83, respectively), EQ VAS (9.34 points; 95% CI, -0.6 to 19.3 points; P = .07 and 13.7 points; 95% CI, -1.7 to 29.0 points; P = .09, respectively) and EQ Index (0.001 point; 95% CI, -0.1 to 0.1 point; P = .98 and 0.08 points; 95% CI, 0-0.2 points; P = .13, respectively). At months 3 and 5, 42% and 47% of patie","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":" ","pages":"966-976"},"PeriodicalIF":20.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11385055/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141889264","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Apixaban vs Aspirin in Patients With Cancer and Cryptogenic Stroke: A Post Hoc Analysis of the ARCADIA Randomized Clinical Trial.","authors":"Babak B Navi, Cenai Zhang, Benjamin Miller, Mary Cushman, Scott E Kasner, Mitchell S V Elkind, David L Tirschwell, W T Longstreth, Richard A Kronmal, Morin Beyeler, Jordan Elm, Richard M Zweifler, Joseph Tarsia, Carlo W Cereda, Giovanni Bianco, Gianluca Costamagna, Patrik Michel, Joseph P Broderick, David J Gladstone, Hooman Kamel, Christopher Streib","doi":"10.1001/jamaneurol.2024.2404","DOIUrl":"10.1001/jamaneurol.2024.2404","url":null,"abstract":"<p><strong>Importance: </strong>Approximately 10% to 15% of ischemic strokes are associated with cancer; cancer-associated stroke, particularly when cryptogenic, is associated with high rates of recurrent stroke and major bleeding. Limited data exist on the safety and efficacy of different antithrombotic strategies in patients with cancer and cryptogenic stroke.</p><p><strong>Objective: </strong>To compare apixaban vs aspirin for the prevention of adverse clinical outcomes in patients with history of cancer and cryptogenic stroke.</p><p><strong>Design, setting, and participants: </strong>Post hoc analysis of data from 1015 patients with a recent cryptogenic stroke and biomarker evidence of atrial cardiopathy in the Atrial Cardiopathy and Antithrombotic Drugs in Prevention After Cryptogenic Stroke (ARCADIA) trial, a multicenter, randomized, double-blind clinical trial conducted from 2018 to 2023 at 185 stroke centers in North America. Data analysis was performed from October 15, 2023, to May 23, 2024.</p><p><strong>Exposures: </strong>Oral apixaban, 5 mg (or 2.5 mg if criteria met), twice daily vs oral aspirin, 81 mg, once daily. Subgroups of patients with and without cancer at baseline were examined.</p><p><strong>Main outcomes and measures: </strong>The primary outcome for this post hoc analysis was a composite of major ischemic or major hemorrhagic events. Major ischemic events were recurrent ischemic stroke, myocardial infarction, systemic embolism, and symptomatic deep vein thrombosis or pulmonary embolism. Major hemorrhagic events included symptomatic intracranial hemorrhage and any major extracranial hemorrhage.</p><p><strong>Results: </strong>Among 1015 participants (median [IQR] age, 68 [60-76] years; 551 [54.3%] female), 137 (13.5%) had a history of cancer. The median (IQR) follow-up was 1.5 (0.6-2.5) years for patients with history of cancer and 1.5 (0.6-3.0) years for those without history of cancer. Participants with history of cancer, compared with those without history of cancer, had a higher risk of major ischemic or major hemorrhagic events (hazard ratio [HR], 1.73; 95% CI, 1.10-2.71). Among those with history of cancer, 8 of 61 participants (13.1%) randomized to apixaban and 16 of 76 participants (21.1%) randomized to aspirin had a major ischemic or major hemorrhagic event; however, the risk was not significantly different between groups (HR, 0.61; 95% CI, 0.26-1.43). Comparing participants randomized to apixaban vs aspirin among those with cancer, events included recurrent stroke (5 [8.2%] vs 9 [11.8%]), major ischemic events (7 [11.5%] vs 14 [18.4%]), and major hemorrhagic events (1 [1.6%] vs 2 [2.6%]).</p><p><strong>Conclusions and relevance: </strong>Among participants in the ARCADIA trial with history of cancer, the risk of major ischemic and hemorrhagic events did not differ significantly with apixaban compared with aspirin.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov Identifier: NCT03192215.</p>","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":" ","pages":"958-965"},"PeriodicalIF":20.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11320331/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141916691","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Substantia Nigra Pathology, Contact Sports Play, and Parkinsonism in Chronic Traumatic Encephalopathy.","authors":"Jason W Adams, Daniel Kirsch, Samantha M Calderazzo, Fatima Tuz-Zahra, Yorghos Tripodis, Jesse Mez, Michael L Alosco, Victor E Alvarez, Bertrand R Huber, Caroline Kubilus, Kerry A Cormier, Raymond Nicks, Madeline Uretsky, Evan Nair, Eva Kuzyk, Nurgul Aytan, Jonathan D Cherry, John F Crary, Daniel H Daneshvar, Christopher J Nowinski, Lee E Goldstein, Brigid Dwyer, Douglas I Katz, Robert C Cantu, Robert A Stern, Ann C McKee, Thor D Stein","doi":"10.1001/jamaneurol.2024.2166","DOIUrl":"10.1001/jamaneurol.2024.2166","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Importance: &lt;/strong&gt;Parkinsonism is associated with traumatic brain injury and chronic traumatic encephalopathy (CTE), a neurodegenerative disease associated with repetitive head impact (RHI) exposure, but the neuropathologic substrates that underlie parkinsonism in individuals with CTE are yet to be defined.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;To evaluate the frequency of parkinsonism in individuals with CTE and the association of RHI and neuropathologic substrates with parkinsonism in these individuals.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Design, setting, and participants: &lt;/strong&gt;This cross-sectional study included brain donors with neuropathologically diagnosed CTE without other significant neurodegenerative disease and with information on parkinsonism from the Understanding Neurologic Injury and Traumatic Encephalopathy brain bank between July 2015 and May 2022.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Exposure: &lt;/strong&gt;Years of contact sports participation as a proxy for RHI.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Main outcomes and measures: &lt;/strong&gt;The main outcomes were frequency of parkinsonism in individuals with CTE and associations between (1) RHI with substantia nigra (SN) Lewy bodies (LBs) and neurofibrillary tangles (NFTs); (2) LBs, NFTs, and arteriolosclerosis with SN neuronal loss; and (3) SN neuronal loss, LBs, NFTs, and arteriolosclerosis with parkinsonism, tested by age-adjusted logistic regressions.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Of 481 male brain donors with neuropathologically diagnosed CTE, parkinsonism occurred frequently in individuals with CTE (119 [24.7%]; 362 [75.3%] did not have parkinsonism). Participants with parkinsonism had a higher mean (SD) age at death (71.5 [13.0] years) than participants without parkinsonism (54.1 [19.3] years) (P &lt; .001) and higher rates of dementia (104 [87.4%] vs 105 [29.0%]), visual hallucinations (45 [37.8%] vs 51 [14.1%]), and probable rapid eye movement sleep behavior disorder (52 [43.7%] vs 58 [16.0%]) (P &lt; .001 for all). Participants with parkinsonism had a more severe CTE stage (eg, stage IV: 35 [29.4%] vs 39 [10.8%]) and nigral pathology than those without parkinsonism (NFTs: 50 of 117 [42.7%] vs 103 of 344 [29.9%]; P = .01; neuronal loss: 61 of 117 [52.1%] vs 59 of 344 [17.1%]; P &lt; .001; and LBs: 28 of 116 [24.1%] vs 20 of 342 [5.8%]; P &lt; .001). Years of contact sports participation were associated with SN NFTs (adjusted odds ratio [AOR], 1.04; 95% CI, 1.00-1.07; P = .03) and neuronal loss (AOR, 1.05; 95% CI, 1.01-1.08; P = .02). Nigral neuronal loss (AOR, 2.61; 95% CI, 1.52-4.47; P &lt; .001) and LBs (AOR, 2.29; 95% CI, 1.15-4.57; P = .02) were associated with parkinsonism. However, SN neuronal loss was associated with SN LBs (AOR, 4.48; 95% CI, 2.25-8.92; P &lt; .001), SN NFTs (AOR, 2.51; 95% CI, 1.52-4.15; P &lt; .001), and arteriolosclerosis (AOR, 2.27; 95% CI, 1.33-3.85; P = .002). In American football players, regression analysis demonstrated that SN NFTs and neuronal loss mediated the association between years of play and parkinsonism in th","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":" ","pages":"916-924"},"PeriodicalIF":20.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11250391/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141616467","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Slowing Down-A Family's Experience With ALS.","authors":"Andrea M Shamaskin-Garroway, Joel Shamaskin","doi":"10.1001/jamaneurol.2024.1922","DOIUrl":"10.1001/jamaneurol.2024.1922","url":null,"abstract":"","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":" ","pages":"907-908"},"PeriodicalIF":20.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141442681","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Centers for Medicare and Medicaid Services Coverage of Amyloid PET.","authors":"Joshua D Grill, Jennifer H Lingler","doi":"10.1001/jamaneurol.2024.2100","DOIUrl":"10.1001/jamaneurol.2024.2100","url":null,"abstract":"","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":" ","pages":"903-904"},"PeriodicalIF":20.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141616466","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prevalence of Epilepsy in People of Sexual and Gender Minoritized Groups.","authors":"Emily L Johnson, Esther Bui, Karina Tassiopoulos, Maya Overby Koretzky, Rodrigo Zepeda, Ernesto Gonzalez-Giraldo, Rebecca F Gottesman","doi":"10.1001/jamaneurol.2024.2243","DOIUrl":"10.1001/jamaneurol.2024.2243","url":null,"abstract":"<p><strong>Importance: </strong>Epilepsy is a highly treatable condition for many people, but there are large treatment gaps with suboptimal seizure control in minoritized groups. The sexual and gender minority (SGM) community is at risk for health disparities, yet the burden of epilepsy in this community is not known.</p><p><strong>Objective: </strong>To estimate the prevalence of active epilepsy among SGM people in the United States.</p><p><strong>Design, setting, and participants: </strong>This was a cross-sectional, nationally representative survey study of community-dwelling US adults who answered questions about epilepsy, sexual orientation, and gender identity in the 2022 National Health Interview Survey (NHIS).</p><p><strong>Exposure: </strong>Self-identification of transgender or gender-diverse identity, or sexual orientation including gay, lesbian, bisexual, or other orientation, excluding straight (ie, heterosexual).</p><p><strong>Main outcomes and measures: </strong>Participants self-reported epilepsy status, medical treatment, seizure frequency, demographic characteristics, sexual orientation, and gender identity. Logistic regression was used to estimate the association of epilepsy with SGM identification.</p><p><strong>Results: </strong>A total of 27 624 participants (15 050 [54%] women; 3231 [12%] Black; mean [SD] age, 48.2 [18.5] years) completed the NHIS and were included. Active epilepsy was present in 1.2% (95% CI, 1.0%-1.3%) of the population. A higher proportion of SGM adults than non-SGM adults reported active epilepsy (2.4% [95% CI, 1.4%-3.3%] vs 1.1% [95% CI, 1.0%-1.3%], respectively). After adjusting for age, race, ethnicity, income, and education, SGM people were more than twice as likely to report active epilepsy than were non-SGM adults (adjusted odds ratio, 2.14; 95% CI, 1.35-3.37).</p><p><strong>Conclusions and relevance: </strong>The findings suggest that SGM adults in the United States have a disproportionate prevalence of epilepsy. The reasons for this disparity are likely complex and may be associated with biological and psychosocial determinants of health unique to this population; as such, these individuals are in need of protected access to medical care.</p>","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":" ","pages":"996-999"},"PeriodicalIF":20.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11264076/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141748175","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Circadian Motor Improvement in MS and Core Body Temperature Dipping.","authors":"Milan Nigam, Alain Créange, Smaranda Leu-Semenescu","doi":"10.1001/jamaneurol.2024.1812","DOIUrl":"10.1001/jamaneurol.2024.1812","url":null,"abstract":"","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":" ","pages":"1000-1001"},"PeriodicalIF":20.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141442678","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Fecal Microbiota Transplantation for Treatment of Parkinson Disease: A Randomized Clinical Trial.","authors":"Filip Scheperjans, Reeta Levo, Berta Bosch, Mitja Lääperi, Pedro A B Pereira, Olli-Pekka Smolander, Velma T E Aho, Nora Vetkas, Lotta Toivio, Veera Kainulainen, Tatyana D Fedorova, Perttu Lahtinen, Rebekka Ortiz, Valtteri Kaasinen, Reetta Satokari, Perttu Arkkila","doi":"10.1001/jamaneurol.2024.2305","DOIUrl":"10.1001/jamaneurol.2024.2305","url":null,"abstract":"<p><strong>Importance: </strong>Dysbiosis has been robustly demonstrated in Parkinson disease (PD), and fecal microbiota transplantation (FMT) has shown promising effects in preclinical PD models.</p><p><strong>Objective: </strong>To assess the safety and symptomatic efficacy of colonic single-dose anaerobically prepared FMT.</p><p><strong>Design, setting, and participants: </strong>This was a double-blind, placebo-controlled, randomized clinical trial conducted between November 2020 and June 2023 with a follow-up period of 12 months at 4 hospitals in Finland. Patients with PD aged 35 to 75 years in Hoehn & Yahr stage 1-3 with a mild to moderate symptom burden and dysbiosis of fecal microbiota were included. Of 229 patients screened, 48 were randomized and 47 received the intervention. One patient discontinued due to worsening of PD symptoms. Two further patients were excluded before analysis and 45 were included in the intention-to-treat analysis.</p><p><strong>Intervention: </strong>Participants were randomized in a 2:1 ratio to receive FMT or placebo via colonoscopy.</p><p><strong>Main outcomes and measures: </strong>The primary end point was the change of Movement Disorder Society Unified Parkinson's Disease Rating Scale parts I-III (part III off medication) at 6 months. Safety was assessed by recording adverse events (AEs).</p><p><strong>Results: </strong>The median (IQR) age was 65 (52.5-70.0) years in the placebo group and 66 (59.25-69.75) years in the FMT group; 9 (60.0%) and 16 (53.3%) patients were male in the placebo group and the FMT group, respectively. The primary outcome did not differ between the groups (0.97 points, 95% CI, -5.10 to 7.03, P = .75). Gastrointestinal AEs were more frequent in the FMT group (16 [53%] vs 1 [7%]; P = .003). Secondary outcomes and post hoc analyses showed stronger increase of dopaminergic medication and improvement of certain motor and nonmotor outcomes in the placebo group. Microbiota changes were more pronounced after FMT but differed by donor. Nevertheless, dysbiosis status was reversed more frequently in the placebo group.</p><p><strong>Conclusions and relevance: </strong>FMT was safe but did not offer clinically meaningful improvements. Further studies-for example, through modified FMT approaches or bowel cleansing-are warranted regarding the specific impact of donor microbiota composition and dysbiosis conversion on motor and nonmotor outcomes as well as medication needs in PD.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov Identifier: NCT04854291.</p>","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":" ","pages":"925-938"},"PeriodicalIF":20.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11287445/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141788033","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Safety and Efficacy of Staged, Bilateral Focused Ultrasound Thalamotomy in Essential Tremor: An Open-Label Clinical Trial.","authors":"Michael G Kaplitt, Vibhor Krishna, Howard M Eisenberg, W Jeffrey Elias, Pejman Ghanouni, Gordon H Baltuch, Ali Rezai, Casey H Halpern, Brian Dalm, Paul S Fishman, Vivek P Buch, Shayan Moosa, Harini Sarva, Ann Marie Murray","doi":"10.1001/jamaneurol.2024.2295","DOIUrl":"10.1001/jamaneurol.2024.2295","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Importance: &lt;/strong&gt;Unilateral magnetic resonance-guided focused ultrasound ablation of ventralis intermedius nucleus of the thalamus for essential tremor reduces tremor on 1 side, but untreated contralateral or midline symptoms remain limiting for some patients. Historically, bilateral lesioning produced unacceptable risks and was supplanted by deep brain stimulation; increasing acceptance of unilateral focused ultrasound lesioning has led to interest in a bilateral option.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;To evaluate the safety and efficacy of staged, bilateral focused ultrasound thalamotomy.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Design, setting, and participants: &lt;/strong&gt;This prospective, open-label, multicenter trial treated patients with essential tremor from July 2020 to October 2021, with a 12-month follow-up, at 7 US academic medical centers. Of 62 enrolled patients who had undergone unilateral focused ultrasound thalamotomy at least 9 months prior to enrollment, 11 were excluded and 51 were treated. Eligibility criteria included patient age (22 years and older), medication refractory, tremor severity (Clinical Rating Scale for Tremor [CRST] part A score ≥2 for postural or kinetic tremor), and functional disability (CRST part C score ≥2 in any category).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Intervention: &lt;/strong&gt;A focused ultrasound system interfaced with magnetic resonance imaging allowed real-time alignment of thermography maps with anatomy. Subthreshold sonications allowed target interrogation for efficacy and off-target effects before creating an ablation.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Main outcomes and measures: &lt;/strong&gt;Tremor/motor score (CRST parts A and B) at 3 months for the treated side after treatment was the primary outcome measure, and secondary assessments for efficacy and safety continued to 12 months.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;The mean (SD) population age was 73 (13.9) years, and 44 participants (86.3%) were male. The mean (SD) tremor/motor score improved from 17.4 (5.4; 95% CI, 15.9-18.9) to 6.4 (5.3; 95% CI, 4.9 to 7.9) at 3 months (66% improvement in CRST parts A and B scores; 95% CI, 59.8-72.2; P &lt; .001). There was significant improvement in mean (SD) postural tremor (from 2.5 [0.8]; 95% CI, 2.3 to 2.7 to 0.6 [0.9]; 95% CI, 0.3 to 0.8; P &lt; .001) and mean (SD) disability score (from 10.3 [4.7]; 95% CI, 9.0-11.6 to 2.2 [2.8]; 95% CI, 1.4-2.9; P &lt; .001). Twelve participants developed mild (study-defined) ataxia, which persisted in 6 participants at 12 months. Adverse events (159 of 188 [85%] mild, 25 of 188 [13%] moderate, and 1 severe urinary tract infection) reported most commonly included numbness/tingling (n = 17 total; n = 8 at 12 months), dysarthria (n = 15 total; n = 7 at 12 months), ataxia (n = 12 total; n = 6 at 12 months), unsteadiness/imbalance (n = 10 total; n = 0 at 12 months), and taste disturbance (n = 7 total; n = 3 at 12 months). Speech difficulty, including phonation, articulation, and dysphagia, were generally mild (rated as not clinically sig","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":" ","pages":"939-946"},"PeriodicalIF":20.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11287440/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141788036","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}